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Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

NCT ID: NCT00076557

Last Updated: 2007-04-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-01-31

Brief Summary

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In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection, and, to date, 6 subjects have been treated with AAV vector in the current hemophilia B liver trial. Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date. In this study, AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel (called the proper hepatic artery or the right hepatic artery).

Detailed Description

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Conditions

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Hemophilia B

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Adeno-Associated Viral with Human Factor IX

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

* Males with severe hemophilia B with Factor IX activity level \< 1% of normal.
* Life expectancy of \> 1 year.
* Age \> 18 years old.
* Ability to give informed consent.
* Greater than twenty exposure days of treatment with Factor IX protein.
* No history or presence of an inhibitor to Factor IX protein.
* Subjects must be able to receive Factor IX protein on a home infusion protocol.
* Subjects must have a normal protime (PT).
* Hepatitis C infected subjects will be evaluated for liver fibrosis based on liver biopsy data graded on a scale of 0-4 (Poynard et. al., 1997). Subjects who are Hepatitis C antibody and RNA positive and have not had a liver biopsy within the last 36 months will be required to have one.
* Subjects must have low AAV titer.
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Stanford University

OTHER

Sponsor Role collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role collaborator

The Hemophilia Center of Western Pennsylvania

OTHER

Sponsor Role collaborator

University of Washington

OTHER

Sponsor Role collaborator

The University of Texas Health Science Center, Houston

OTHER

Sponsor Role collaborator

University of Campinas, Brazil

OTHER

Sponsor Role collaborator

Christian Medical College, Vellore, India

OTHER

Sponsor Role collaborator

Royal Prince Alfred Hospital, Sydney, Australia

OTHER

Sponsor Role collaborator

Avigen

INDUSTRY

Sponsor Role lead

Locations

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Stanford University

Palo Alto, California, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

The Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Site Status

Countries

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United States

Related Links

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http://www.avigen.com/research.php

Avigen Ongoing Clinical Trials

Other Identifiers

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BB IND 9398

Identifier Type: -

Identifier Source: org_study_id